Year In Review: Gene Therapies Make a Big Splash in 2023

Gene therapies are changing the way we treat diseases by targeting the cause of a disease directly at the DNA level, rather than mitigating symptoms, and creating long-lasting change in the body. 2023 was a pivotal year in this field with several gene therapies approved to help patients and families battling Duchenne muscular dystrophy, amyotrophic lateral sclerosis, hematologic malignancies, Type 1 diabetes, and to help adults with severe hemophilia A. And still, we could see the first FDA-approved gene therapy that aims to cure sickle-cell disease and transfusion-dependent β-thalassemia before the year ends.

As many more therapies advance through the pipeline, IGT continues to advocate for modernized policies that will help clear the barrier-ridden pathway to ensure these treatments can get to patients who need them. And because these treatments will not only help patients thrive but will also help deliver value to our healthcare system, IGT worked to demonstrate this value to key stakeholders throughout this year.

Take a look at some 2023 highlights below:

Congressional Briefing on the Science of Gene Therapy and Policy Priorities: IGT hosted a panel discussion on the science of gene therapy, its impact on patients and families, and the role of Congress in helping to ensure patient can benefit from these transformational therapies.

Value Principles Launch: IGT developed critical principles and related elements of value, identifying the pillars that continue to guide our efforts to modernize policies that bolster innovation, improve patient access, ensure coverage, and ultimately account for the true value of these transformative therapies. More from Chairman Erik Paulsen, here.

Patient Perspective: Nate Plasman, whose son was diagnosed with Duchenne muscular dystrophy, penned a blog on the value of gene therapies and the hope they provide to patients and families.

Legislative Win: Representatives Brett Guthrie (R-KY), Anna Eshoo (D-CA), Mariannette Miller-Meeks (R-IA), Jake Auchincloss (D-MA), John Joyce (R-PA) and Scott Peters (D-CA) introduced the Medicaid VBPs for Patients Act, or the MVP Act (H.R. 2666).

• IGT Chairman Erik Paulsen released a statement applauding this important bill.

• IGT Patient Advocacy Advisory Council member, Lauren Holder penned an op-ed in The Hill reiterating the critical need for the MVP Act.

• Several other stakeholders expressed support for the MVP Act here.

Congressional Briefing on Access Issues: IGT hosted a briefing featuring patients and advocates in the sickle cell patient community to discuss the many access issues patients face and policy solutions to help ensure patients may benefit from current and future treatments.

Reviving the Personalized Medicine Caucus: IGT Chairman Erik Paulsen was instrumental in John Moolenaar (R-MI) becoming a Co-Chair of the Caucus, largely due to his interest in gene therapy access issues that overlap with the PMC’s mission.

Expanding the Patient Advocacy Advisory Council: IGT added three new members to its PAAC, which focus on advocacy issues surrounding Duchenne muscular dystrophy and Huntington’s Disease.

• Laura McLinn, Founder of the Best Day Ever Foundation

• Parent Project Muscular Dystrophy

• Lauren Holder, Huntington’s Disease Patient Advocate and Producer/Host of Help 4 HD Live

• Children’s Sickle Cell Foundation, Inc.

Engagement with Key Stakeholders: Throughout 2023, IGT leaders had the opportunity to participate in several panel discussions, podcasts, and events to showcase the value of gene therapies and the work being done to advance policies that will support their development, including:

• EveryLife Foundation’s 2023 Newborn Screening Bootcamp

• The American Society of Cell and Gene Therapy 2023 policy summit

• The Alliance for Regenerative Medicines Congressional Briefing

• Rally’s healthcare panel on Gene Therapy: Breakthroughs in Genetic Medicine

• Artia Solutions’ Crossroads Conference

• Barnes and Thornburg’s Panel on Life Sciences Lifeline - The Promise And Challenges Of Gene Therapy during the Indiana Health Industry Forum’s Annual Conference

• The Help for HD Live Podcast

• The National Minority Quality Forum Leadership Summit

• PTC Bio’s webinar “Rare Disease in the Black Community”

• Sickle Cell Disease Association of America’s Annual National Convention

Regulatory Updates: 2023 also provided several opportunities to weigh in on key proposed policy and regulatory updates, including:

• Discrimination on the Basis of Disability in Health and Human Service Programs or Activities Proposed Rule [HHS-OCR-2023-0013]

• Medicaid Program; Misclassification of Drugs, Program Administration and Program Integrity Updates Under the Medicaid Drug Rebate Program [CMS-2434-P]

• FY 2024 IPPS Proposed Rule [CMS-1785-P]

• EEOC Draft Strategic Enforcement Plan for FYs 2023-2027 [EEOC-2022-0006]

IGT will continue efforts to update our healthcare system to clear the way for new transformative gene therapies now and in the future. Patients, families, and our healthcare ecosystem stand to benefit greatly.